RecruitingOBSERVATIONAL

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

The Rocket Study is looking at boys aged 6 months to 17 years who have a rare genetic condition called Pelizaeus-Merzbacher Disease (PMD). Doctors want to learn more about how PMD progresses and changes in the body. They will be looking at certain markers in fluid from around the brain and spinal cord, as well as in blood, and using brain scans. They'll also talk to participants and their parents/carers about how the disease affects their daily lives. All this information will help researchers create new treatments for PMD in the future. It's a study where participants are observed, not given new medicines.

At a glance

Status

Recruiting

Sponsor

Ionis Pharmaceuticals, Inc.

Enrolment target

Start

03 Oct 2022

Estimated completion

01 Mar 2029

Pelizaeus-Merzbacher Disease

What is this study about?

The Rocket Study is an important research project for boys with a rare genetic condition called Pelizaeus-Merzbacher Disease (PMD). PMD affects the brain and nervous system, and there isn't a cure yet. This study is all about understanding the disease better, which is a key step towards finding new treatments.

Researchers want to see how PMD changes over time in different ways. They will be looking for specific 'markers' in special fluids from the body, like the fluid that surrounds the brain and spinal cord (often called CSF), and in blood samples. They will also use special brain scans. On top of that, they will ask participants, their families, and carers about how PMD impacts their life and day-to-day activities.

By gathering all this information, researchers hope to get a clearer picture of how PMD works. This better understanding will help them design and test new medicines or therapies that could make a real difference for people with PMD in the future. It’s important to remember this study focuses on understanding the disease, not on trying out new medications.

Key takeaways

The study aims to understand how Pelizaeus-Merzbacher Disease (PMD) affects the body over time.
It involves boys aged 6 months to 17 years with a specific genetic change in the PLP1 gene.
Researchers will collect samples (blood, CSF) and perform brain scans to gather information.
No new treatments or medications will be given during this study.
The information gained could help develop new PMD treatments in the future.
Participation lasts about 26 months and requires multiple visits.

Who may be eligible?

This study is particularly looking for boys who are diagnosed with Pelizaeus-Merzbacher Disease (PMD).

To be considered for the study, boys must be between 6 months and 17 years old. They also need to have a confirmed genetic test result showing they have a specific change (a duplication) in a gene called PLP1, which is known to cause PMD. Their parents or carers must be able to give written permission and attend all study appointments, as well as share information about the boy's health and how PMD affects him.

There are also a few reasons why someone might not be able to join. For example, if they have certain health problems that make it unsafe to have some of the study checks, or if they have more than two copies of the PLP1 gene. The study doctor will carefully check if it's suitable for each boy to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is the person with PMD male?
Are they between 6 months and 17 years old?
Do they have a confirmed diagnosis of PMD caused by a specific PLP1 gene duplication?
Is a parent or carer able to attend all study visits and provide feedback?
Are there no medical reasons preventing blood tests, spinal fluid tests, or brain scans?

Answer every question to see your result.

What does participation involve?

If you decide to take part in the Rocket Study, you would be involved for about 26 months, which is just over two years. During this time, you would have several visits where doctors would collect specific information.

These visits would include some medical checks, collecting blood samples, and having a special procedure called a lumbar puncture to collect fluid from around the spinal cord (CSF). You would also have brain scans (neuroimaging). If needed, some of these procedures could be done while you are gently sedated or under a general anaesthetic to make them more comfortable. You would also be asked to complete some assessments about how the disease affects you and your daily life, and your parents or carers would provide information too. You would not be given any new medications as part of this study.

Potential risks and benefits

Taking part in this study won't directly improve your PMD, as it's designed to gather information, not to offer a new treatment. However, the information collected could benefit future patients by helping scientists develop new treatments for PMD. There are some small risks involved, such as discomfort from blood tests, lumbar punctures (where a needle collects fluid from the spine), and brain scans, or from sedation if it's used. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (9)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Ionis Investigative Site
Verified postcode
Atlanta, United States· Recruiting
Ionis Investigative Site
Verified postcode
Philadelphia, United States· Recruiting
Ionis Investigative Site
Verified postcode
Clermont-Ferrand, France· Active not recruiting
Ionis Investigative Site
Verified postcode
Le Kremlin-Bicêtre, France· Active not recruiting
Ionis Investigative Site
Verified postcode
Göttingen, Germany· Active not recruiting
Ionis Investigative Site
Verified postcode
Tel Aviv, Israel· Recruiting
Ionis Investigative Site
Verified postcode
Milan, Italy· Recruiting
Ionis Investigative Site
Verified postcode
Amsterdam, Netherlands· Recruiting
Ionis Investigative Site
Verified postcode
Leeds, United Kingdom· Recruiting

Common questions

What is Pelizaeus-Merzbacher Disease (PMD)?

PMD is a rare genetic condition that affects the brain and nervous system, leading to problems with movement and development.

Will my child receive new treatment in this study?

No, this study is about understanding PMD better, not about testing new treatments. Participants will not receive any new medications.

What is a lumbar puncture?

A lumbar puncture is a medical procedure where a small amount of fluid from around the spinal cord (CSF) is collected using a thin needle. It's done to get important information about the brain and nervous system.

How long will my child be in the study?

Each child will be involved in the study for about 26 months, or just over two years.

Is this study only for boys?

Yes, this specific study is looking for male participants between 6 months and 17 years old.

How to find out more

Ionis Pharmaceuticals

IonisPelizaeusMerzbacherStudy@clinicaltrialmedia.com (844) 430-1848 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.